Marijke van den Berg, MD, World Federation of Hemophilia (WFH) Vice President, Medical, opened the WFH President’s Address by welcoming attendees and introducing Alain Weill, President of the WFH.
The focus of the WFH President’s Address was two Calls to Action. The first Call to Action acknowledged the urgent need to develop a diagnostic device that is reliable, easy to use and affordable. Hemophilia needs something as simple as the skin prick test for glucose monitoring.
Weill described disparities between the developing and developed worlds in terms of the amount of factor VIII available and the degree of underdiagnosis. Fifty-five countries have less than one international unit per capita. In 23 countries, less than 10 per cent of people with hemophilia receive a diagnosis. “This is neither fair nor acceptable,” said Weill, “and we need to keep fighting to improve this situation.”
Weill went on to discuss the global supply of clotting factor concentrates (CFCs). Approximately two-thirds of worldwide factor VIII supplies (11 billion international units per year) are used by 30% of the global hemophilia A population; and these people predominantly live in Europe, North America. It would take a further 29 billion international units per year to provide the remaining 70% hemophilia A population with the same access to treatment. The pharmaceutical industry sold approximately 12 billion international units of factor VIII in 2016; therefore, factor VIII production would have to increase by 270% to meet the needs of the global hemophilia A community. This calculation assumes a 1% factor trough level is needed. “What if we aim for a 5% factor trough level? We would need over 150 billion international units. Are either of these scenarios possible?” said Weill.
This brought Weill to his second Call to Action focused on making a commitment to increasing factor concentrate production, ensuring gene therapy is accessible to all regardless of income, and improving the accessibility and affordability of non-substitutional therapies. “Although some companies have stepped up and donated factor concentrates, many more companies should be doing this,” said Weill. “Another priority,” said Weill, “is to track the vast volumes of unused cryoprecipitate as it can be used to make factor concentrates.” While these issues can be addressed relatively quickly, other priorities, such as creating new manufacturing facilities for extended half-life products and increasing access to non-substitutional therapies may take longer. Finally, he implored pharmaceutical companies to not allow legal feuds hinder access to innovative treatment.
“Moving forward on these issues will require the willingness and support of governments,” said Weill. “Hemophilia and all other inherited bleeding disorders have to be included in health policies in every country, especially in countries where diagnosis and treatment are well below standard of care.”
Weill concluded his talk by proposing that future WFH Board of Directors work with all relevant stakeholders, such as clinicians, industry representatives, health economists, laboratory technicians, and people with hemophilia, to submit realistic proposals at the next WFH Congress to address these Calls to Action.